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A Parkinson’s Drug Moves Forward With Help From Michael J.Fox


(Forbes) Today, at a medical conference being hosted by the Michael J. Fox Foundation, a biotechnology company is presenting the first human data from a Parkinson’s drug that several of the world’s largest drug companies nearly abandoned.


The company, Denali Therapeutics, is run by a widely respected team that formerly worked at Genentech, the Roche unit, and it had one of the largest biotech initial public offerings of 2017. But the stock is down 16% from its IPO price.


Denali’s drug targets a protein called LRRK2 that is involved in cells’ trash-disposal systems. The gene that makes it is the most common genetic cause of Parkinson’s. The LRRK2 gene variant was discovered 15 years ago. Sergey Brin famously carries a copy of it. 23andMe, the consumer genetics startup, has studied it. Pfizer, Merck and Genentech were all working on drugs targeting the gene when it was discovered that those drugs seemed to cause worrisome lung changes in animals. They all stopped work on it. At that point, this promising genetic hint to a Parkinson’s treatment looked as if it would never be investigated.


Then the Fox Foundation stepped in, organizing meetings at which all the companies developing LRKK2 drugs pooled their results and tried to understand what might have gone wrong. The team at Genentech — now the team at Denali — was part of those meetings, and they started their own company and licensed the Genentech molecule. That’s the drug on which data is being presented today. Read More



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